移植自體同源的幹細胞可望治療紅斑狼瘡

伴有嚴重肺臟損害的紅斑狼瘡女患者可以獲得21個月症狀緩解期

  2002年6月7日報導─據《Arthritis & Rheumatism》雜誌6月份一期中一項單個病例的報導,自體同源性血液幹細胞的移植,可能可以有效治療系統性紅斑狼瘡(SLE)。在移植CD34+細胞後進行一段免疫抑制的治療後,一位伴有嚴重肺臟損傷且對藥物治療無效的女性患者,可以在21個月內不用任何的藥物治療,而且肺臟和腎臟的功能得到了恢復。

  奧地利維也納大學的M. Brunner醫師,和他的同事們寫道:「對於患有嚴重自身免疫性疾病的患者包括SLE,利用移植自身同源性血液幹細胞的方法,來清除免疫系統中自身免疫性T細胞是一種治療的選擇。」

  儘管在4年的時間堙A採用了類固醇、azathioprine、cyclophosphamide、和immunopheresis的強化治療,這名18歲的女性仍患有涉及機體多個系統的SLE並需要接受重覆的機械性通氣來治療狼瘡性肺炎。在調整了cyclophosphamide和antithymocyte globulin的用量後,她接受了每公斤體重8.87 x 106純化的CD34+細胞的注射。在治療期間,她出現了發熱和敗血症,接著出現了帶狀皰疹。

  在為期9天的治療中,患者出現了造血功能的恢復,並且在接下來的21個月中,她處於完全的臨床恢復期,伴有肺臟和腎臟功能的恢復,並且免疫抑制劑停用後,其SLE的標誌為陰性。

  作者寫道:「儘管仍需要更長時間的隨訪,來評價這種治療效果的持久程度,但是患者的治療過程提示,自身同源性血液幹細胞的移植,可能是患有SLE患者再次喚起免疫耐受的一種方法。對於高危險患者的診斷水準提高,將有可能在疾病的早期階段,在出現嚴重的終末器官損害之前實施〔移植〕。」

  Brunner的研究小組和其他小組正在設計控制臨床對SLE患者進行自身同源性血液幹細胞移植試驗的方法。

Autologous Blood Cell Transplant Holds Promise in SLE

21-Month Remission in Woman With Severe Pulmonary Impairment

By Laurie Barclay, MD
WebMD Medical News

Reviewed by Gary D. Vogin, MD

June 7, 2002 -- Autologous blood stem cell transplantation may have potential for effectively curing systemic lupus erythematosus (SLE), according to a single case report in the June issue of Arthritis & Rheumatism. Following a course of immunosuppressive therapy with transplant of CD34+ cells, a woman with severe pulmonary impairment refractory to medication has been free of all medications for 21 months, with restored pulmonary and renal function.

"For patients with severe forms of autoimmunity, including SLE, purging autoreactive T cells from the immune repertoire by transplanting autologous hematopoietic stem cells is a therapeutic option," write M. Brunner, MD, and colleagues from the University of Vienna in Austria.

Despite aggressive treatment with steroids, azathioprine, cyclophosphamide, and immunopheresis over a 4 year period, this 18 year-old woman had multisystem involvement with SLE and required repeated mechanical ventilation for lupus pneumonitis. After conditioning with cyclophosphamide and antithymocyte globulin, she received an infusion of 8.87 x 106 purified CD34+ cells per kg of body weight. She had fever and septicemia during treatment and a subsequent episode of herpes zoster.

Within 9 days of treatment there was hematopoietic regeneration, and at 21-month follow-up she continued to be in complete clinical remission, with normal pulmonary and renal function and negative SLE markers off all immunosuppressants.

"Although a longer follow-up is required for assessment of the durability of response, the patient's course indicates that autologous hematopoietic stem cell transplantation may be a way to reinduce tolerance in patients with SLE," the authors write. "Advances in identifying high-risk patients may allow for [transplantation] during an earlier stage of disease, before the development of severe end organ damage."

Brunner's group and others are beginning controlled clinical trials of autologous blood stem cell transplantation in SLE.

© 2002 WebMD Inc. All rights reserved.

    
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